Gene Therapy: Coming Of Age

By Sankalp Sethi, Alexandra Burr, Jedediah Seltzer and Julia Vannoni

The potential promise of gene therapy was evident in 2022, with FDA approvals for both Hemgenix in hemophilia B and Zynteglo in beta thalassemia. However, physician and patient hesitancy continue given the ongoing worrisome headlines related to safety and manufacturing. Many are familiar with the failures during gene therapy’s first entry in the clinic in 1990, which sent the industry into a holding pattern for over ten years. The known complexity of this industry makes the ongoing failure and setbacks less surprising.

Gene therapy is in a period of growth and with that comes challenge and failure. This is not the first time a biologic industry has experienced this. Explore some of the clinical and technical challenges gene therapies face along with the similarities and differences between gene therapy and monoclonal antibodies, including launched asset success, phase-transition failure rates, and more.